Rare Disease Treatment Market - Growth, Trends, Covid-19 Impact, and Forecasts (2022 – 2027)

The Rare Disease Treatment Market is projected to register a CAGR of 8.6% during the forecast period (2022 to 2027).

The healthcare system has witnessed enormous challenges due to the COVID-19 pandemic. All outpatient treatments were postponed or restricted during the COVID-19 pandemic to ​reduce the risk of viral transmission, as most chronic therapies were regarded as non-urgent. A review article titled “Research and Management of Rare Diseases in the COVID-19 Pandemic Era: Challenges and Countermeasures,” published in the journal of Frontiers in Public Health in April 2021, studied the challenges faced by patients affected by rare diseases. The article concluded that regular clinical services, counseling, and therapies were extremely inadequate. Also, the clinicians, researchers, and scientists working on rare genetic diseases face extra challenges due to the pandemic since research projects and clinical trials for rare and genetic diseases were stalled during the COVID-19 pandemic.

The definition of rare diseases has distinct in different parts of the world. The United States Food and Drug Administration defines a rare disease as a disease or condition that affects less than 200,000 people in the United States. The European Commission Public Health states that a rare disease affects no more than one person in 2000 in the European Union. According to Health Canada, the rare disease affects less than five persons in 10,000 in Canada. According to an article published in March 2021, titled “Epidemiology of rare diseases is important”, over 6,000 different rare diseases are thought to exist and more than 70% of rare diseases are hereditary, and the majority of them have a chronic course. According to the February 2022 update of the rarediseaseday.org, about 300 million people around the world are living with rare diseases which is quite a high and thus, the demand for effective and advanced treatment solutions for rare diseases is expected to increase due to the high burden and growing awareness about these diseases which is expected to fuel growth in the studied market over forecast period.

An article in the journal of Diseases in November 2020 titled “Precision Medicine in Rare Diseases” discussed using precision or personalized medicine to treat rare diseases. The study indicated that more than 90% of rare diseases do not have an effective treatment method. This inadequacy in the therapies for rare diseases necessitates novel treatment methods, which is expected to propel the growth of the rare disease treatment market in the forecast period.

Various governments around the world have been formulating several initiatives for the treatment of rare diseases. For instance, the Indian government Ministry of Health and Family Welfare’s National Policy for Rare Diseases, 2021 aimed at lowering the incidence and prevalence of rare diseases based on an integrated and comprehensive preventive strategy encompassing awareness generation, premarital, post-marital, pre-conception, and post-conception screening and counseling programs to prevent births of children with rare diseases, and within the constraints on resources and competing health care priorities, enable access to affordable health care to patients of rare diseases which are amenable to one-time treatment or relatively low-cost therapy.

The increase in the necessity of rare disease treatment worldwide is expected to propel the growth of the rare disease treatment market.​ However, a lack of awareness regarding rare disease treatment and the high cost of treatment is expected to hinder the market growth during the study period.

Key Market Trends

The Biologics Segment is Expected to Hold a Major Market Share in the Rare disease treatment Market.

Biologics occupy a major segment in the rare disease treatment market. Over the years, various market players have invested in researching and developing new biologics for the treatment of rare diseases. For example, CHIESI Farmaceutici S.p.A. in February 2020, Chiesi launched Global Rare Diseases, a new business unit segment focused on the research, development, and commercialization of treatments and patient support services for rare and ultra-rare disorders. In May 2020, Chiesi Global Rare Diseases, together with Protalix BioTherapeutics, announced the submission of a biologics license application to the US Food and Drug Administration for Pegunigalsidase Alfa for the treatment of Fabry disease. Upon approval of the biologics license application, Protalix BioTherapeutics will be eligible for a breakthrough payment from Chiesi to assist in the next phase III study of the drug. Increasing investment in treating rare diseases such as Fabry disease is expected to fuel the growth of the market under study.​

An article titled “Biological Treatments in Inflammatory Bowel Disease: A Complex Mix of Mechanisms and Actions,” published in the MDPI journal of Biologics in August 2021, studied the main biological treatments currently available for inflammatory bowel disease treatment due to the extremely important current role of biologics. The study showed that various biologic therapies such as anti-TNF-α therapy, anti-integrin therapy, anti-cytokine therapy, and other new biologics are widely used to treat rare inflammatory bowel disease. The study indicated that further studies are needed to better understand the action mechanism of the biological drugs, which will help understand how to improve the efficacy and safety of the treatment methods.​

Another article presented in the Orphanet Journal of Rare Diseases titled “Essential list of medicinal products for rare diseases: recommendations from the IRDiRC Rare Disease Treatment Access Working Group,” published in July 2021, studied the list of biologics approved by the United States Food and Drug Administration (FDA), the European Medicines Agency (EMA) and China’s National Medical Products Administration (NMPA), which are useful against the rare diseases. The goal of the list was to further accelerate interactions among patient organizations, health care providers, industry, and government agencies to improve standards of care for rare diseases by promoting access to treatments.

The increasing research on the study of biologics for rare disease treatment is expected to boost the growth of the studied market over the forecast period.

North America is Expected to Hold a Significant Share in the Rare Disease Treatment Market

The North American region is expected to occupy a major share in the rare disease treatment market owing to the high prevalence of the rare diseases in the region such as Huntington's disease, spina bifida, fragile X syndrome, Guillain-Barré syndrome, Crohn's disease, cystic fibrosis, and Duchenne muscular dystrophy, high awareness about these diseases and presence of robust healthcare system for the diagnosis and treatment of rare diseases. For instance, according to the 2021 report of the Canadian Organization for Rare Disorders (CORD), every year about 1 in 12 people in Canada are affected by rare diseases, and about 80% of these diseases are caused due to genetic changes. Hence, owing to the high burden of rare diseases in the region, it is expected to have significant growth over the forecast period.

In the North American region, the United States is expected to have a significant share owing to the high demand for treatment of rare diseases due to high prevalence, ongoing research, and development activities new investments in the area, and other factors. For instance, according to the February 2020 report of the Center for Disease Control and Prevention (CDC) on rare diseases, about 30 million people are affected by approximately 7,000 rare diseases in the country, with the majority of them being children and thus, the demand for effective and advanced therapeutics is very high in the country which is expected to boost growth in the studied market. Further, the research budget for rare diseases in the United States is one of the highest compared to other parts of the world and positive result from these research activities is expected to have a significant impact on the studied market in the United States. For example, as per the May 2022 report of the National Institute of Health of the United States, about USD 6,191 million was spent on rare disease research activities in 2021 and it is expected to increase to USD 6,482 million in 2022. Therefore, owing to the above-mentioned factors, the North American region is expected to grow and occupy a significant share of the studied market.

Competitive Landscape

The Rare disease treatment Market is competitive and consists of several major players. In terms of market share, a few major players dominate the market. Some companies currently dominating the market are Amgen, Inc., Alexion Pharmaceuticals, Inc., Bristol-Myers Squibb Company, Biomarin Pharmaceuticals, Bayer AG, and others. Market players readily adopt multiple initiatives such as mergers and acquisitions and product launches to strengthen their position in the market.

Additional Benefits:

• The market estimate (ME) sheet in Excel format
• 3 months of analyst support